What Is the FDA Expanded Access Program for Patients With Terminal Illness?

The FDA Expanded Access Program, also known as Compassionate Use, is an option that allows patients with terminal illnesses to try investigational medical products as treatments outside of clinical trials if there aren’t any comparable therapies that can be used instead. Investigational medical products are defined as drugs, biologics, or medical devices that are currently being tested to determine whether they are safe and appropriate for use in patient populations. Since these products are being tested, they have not yet been approved by the FDA. As a result, there aren’t any guarantees that the product will be effective in the treatment of the condition, and there is a possibility that using the product may cause adverse reactions. 

Being eligible for expanded access does not always guarantee that a patient will receive an investigational medical product. For example, sometimes there are limited amounts of medications available for compassionate use, such as if a pharmaceutical company only has enough medication for a clinical trial. Additionally, although expanded access is legal, it is limited to specific patients based on criteria. All of the following criteria must be met for expanded access to be deemed an appropriate option: 

• The patient must have a life-threatening or serious disease or condition
• The potential benefit to the patient is significant in comparison to the potential risk 
• It is not possible for the patient to enroll in a clinical trial 
• There isn’t a satisfactory or comparable alternative therapy for the disease or condition 
• Providing the investigational medical product will not interfere with any investigational trials to support product development or marketing approval for treatment indications 

It is important to note that the FDA Expanded Access Program is not the same as the Right to Try Act, which was passed in 2018. Although the Right to Try Act is another option that patients may use to have access to medications that have not yet been approved by the FDA, the eligibility criteria differ from expanded access, and medications that may be provided through Right to Try have already been used in Phase I clinical trials. 

Sources

“Compassionate Drug Use.” American Cancer Society. https://www.cancer.org/cancer/managing-cancer/making-treatment-decisions/clinical-trials/compassionate-drug-use.html 

“Expanded Access.” U.S. Food and Drug Administration. https://www.fda.gov/news-events/public-health-focus/expanded-access#:~:text=Sometimes%20called%20%E2%80%9Ccompassionate%20use%E2%80%9D%2C,trials%20when%20no%20comparable%20or 

“Expanded Access: Information for Patients.” U.S. Food and Drug Administration. https://www.fda.gov/news-events/expanded-access/expanded-access-information-patients